Zitter Pulse Check: Orphan Drugs (June 2023)

Coverage

Orphan drugs is a high-cost category with an increasing number of therapies that treat the conditions. To help stakeholders absorb datapoints and perceptions from all directions and synthesize those insights into a tangible strategy for orphan diseases, Zitter Insights surveys a leading panel of pharmacy and therapeutics (P&T) decision makers at payers and integrated delivery networks. The Managed Care Biologics and Injectables Index: Orphan Drugs combines deep payer insights with the industry standard in market access information to produce quarterly reports and insights summaries on leading specialty brands.

Payer Management

In the U.S., orphan diseases are conditions impacting fewer than 200,000 people. There are more than 7,000 of these rare illnesses affecting an estimated 30 million Americans, and new diseases continue to be discovered. Payers covering almost two-thirds of commercial lives and ones covering more than four-fifths of Medicare beneficiaries said that managing branded non-oncology orphan drugs is a high or very high priority. Commercial payers with almost one-third of lives and Medicare payers representing almost three-fourths of lives said they are somewhat aggressive in their management of these agents.

Payers with almost two-fifths of covered commercial lives said their organization had an average level of contracting for orphan drugs, while those with almost three-fourths of Medicare lives said the same. Payers with almost one-third of commercial beneficiaries and more than a quarter of Medicare lives reported that orphan drugs have a very high budget impact.

Key Findings

Management Strategies

Among payers covering commercial lives, the top social determinants of health initiative for disadvantaged members taking non-oncology orphan drugs is offering greater access to telehealth — which is offered by payers with three-fourths of lives — followed by providing services to assist in health/medicine language barriers and delivering services to support health/medicine literacy. Payers covering more than two-thirds of Medicare lives have partnerships with housing authorities or community organizations to assist members taking orphan drugs, and slightly less provide greater access to telehealth for these beneficiaries.

Social Determinants of Health

Among payers covering commercial lives, the top social determinants of health initiative for disadvantaged members taking non-oncology orphan drugs is offering greater access to telehealth — which is offered by payers with three-fourths of lives — followed by providing services to assist in health/medicine language barriers and delivering services to support health/medicine literacy. Payers covering more than two-thirds of Medicare lives have partnerships with housing authorities or community organizations to assist members taking orphan drugs, and slightly less provide greater access to telehealth for these beneficiaries.

Payer Priorities

Among non-oncology orphan drug categories, payers with almost half of commercial lives said that hemophilia was their most highly prioritized category to manage mainly due to the total cost of care, followed by multiple sclerosis, also due to its high cost of treatment, as well as the large amount of beneficiaries with the condition. Other areas of concern for commercial payers were immunodeficiency, pulmonary arterial hypertension and growth hormone deficiency. Payers with slightly less than half of Medicare lives cited multiple sclerosis as their most highly prioritized orphan drug category due to its high cost of treatment, followed by acromegaly because of competitive contracting. Growth hormone deficiency, hereditary angioedema, hemophilia, anemia and spinal muscular atrophy were other conditions respondents said their organization prioritized in managing Medicare beneficiaries.

Trends

Contracting Strategy

Message: “Ascendis is following up to initial clinical presentation to discuss coverage of Skytrofa. Currently Skytrofa is not covered on formulary. We discussed the growth hormone category as a whole, that our sole preferred is Genotropin. We reviewed Skytrofa dosing and weekly administration and Ascendis saying that multiple providers have expressed interest in having it available for their patients.”

Payer Thoughts: “We see this category as commoditized. We see it mainly as a lifestyle category and non-urgent. We don’t see weekly administration as much of an advantage over three, six or seven doses/week to justify increased cost.”

FDA Approves Biogen ALS Drug Qalsody

In April 2023, the FDA gave accelerated approval to Biogen Inc.’s Qalsody (tofersen) for the treatment of amyotrophic lateral sclerosis (ALS) in adults who have a mutation in the superoxide dismutase 1 (SOD1) gene. The company says it is the first treatment to target a genetic cause of ALS. The agency gave the agent priority review, as well as orphan drug and fast track designations. Biogen is sponsoring a genetic testing program, ALS Identified, that is offered through Invitae that helps with access to genetic counseling and testing for people at least 18 years old within the U.S. and Puerto Rico who have a clinical diagnosis of ALS or a family history of ALS at no charge. According to Biogen, SOD1-ALS is diagnosed in about 2% of ALS cases. The FDA says it estimates that fewer than 500 people in the U.S. are living with SOD1-ALS. The recommended dose is 100 mg via a one-to-three minute intrathecal injection. Initial administration consists of three loading doses at 14-day intervals, and then maintenance dosing is once every 28 days. The price per dose is $14,230.

Subscribers to AIS’s RADAR on Specialty Pharmacy may read the in-depth article online

FDA Approves Hemophilia B Gene Therapy, but Its Cost May Pose Access Problems for Payers

The FDA recently approved the fifth and sixth gene therapies, with four of those decisions coming in the second half of 2022. While payer respondents to a Zitter Insights survey have expressed interest in hemophilia B treatment Hemgenix (etranacogene dezaparvovec-drlb), its price may prove to be an obstacle to coverage.

On Nov. 22, 2022, the FDA approved uniQure N.V.’s Hemgenix for the treatment of people at least 18 years old with hemophilia B who currently use factor IX prophylaxis therapy or have current or historical life-threatening hemorrhage or have repeated serious spontaneous bleeding episodes. CSL Behring LLC, a CSL business, will market the gene therapy. The agency gave the first-in-class adeno-associated virus A5-based gene therapy — which previously was known as EtranaDez — priority review and orphan and breakthrough therapy designations.

Subscribers to AIS’s RADAR on Specialty Pharmacy may read the in-depth article online

0

0

0 Comments

0

0

• Pulse Checks